Gene Therapy
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‘Suffering’: B.C. family desperate to get gene therapy for son with rare conditionNavpreet and Stalin Gill told Global News that they began to notice their now three-year-old son, Gurmoh, having mobility issues around age one.Health7 hours
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FDA approves 2 gene therapies for sickle cell diseaseThe U.S. FDA on Friday approved two gene therapies for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, as well as from bluebird bio.HealthDec 8, 2023
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3 Alberta siblings with rare, terminal disorder finally have hope for a treatmentA family from Alberta with three children who have been diagnosed with the rare and fatal Batten disease has been given a new kind of hope due to an older medication.HealthAug 10, 2023
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Groundbreaking gene therapy gives the gift of sight to Quebec childDoctors at the Maisonneuve Rosemont ophthamology clinic performed groundbreaking surgery on an 11-year-old boy, reversing a rare genetic condition that can cause blindness.CanadaMay 29, 2023
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Ontario family seeks answers, treatment for child’s rare genetic disease'Her birthday is in October. We don't know what's going to be going on then,' said Jonelle Vlogiannitis of her daughter Avery's degenerative disease.HealthAug 16, 2022
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‘Uncharted territory’: Toronto family of child with rare disease raises $3M for therapy'This could change his life,' said Terry Pirovolakis of a gene therapy clinical trial to treat son Michael's ultra-rare condition, SPG50.HealthAug 15, 2022
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Nova Scotia celebrates research milestone for patients with Fabry diseaseNova Scotia is home to some of the world's first patients to receive an experimental gene therapy for treating Fabry disease and the newly-published study results are promising.HealthFeb 28, 2021
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‘He’s the only child living with CLN2 in Saskatchewan’: community rallies for 4-year-old with rare diseaseThe community is rallying behind a four-year-old Regina boy who has been diagnosed with a rare disease, hoping to one day help fund a cure.HealthAug 28, 2019
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First in-body CRISPR study set to start in U.S. to treat form of blindnessThe experimental treatment aims to supply kids and adults with a healthy version of the gene they lack, using a tool that cuts or “edits” DNA in a specific spot. It’s intended as a one-time treatment that permanently alters the person’s native DNA.ScienceJul 25, 2019
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Calgary patient with rare condition ‘first in the world’ to receive experimental gene therapyDoctors in Calgary have used gene therapy, in what is believed to be a world-first, to treat a patient with a rare condition that can damage major organsHealthFeb 16, 2017
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